Joint proposal to facilitate clinical investigation of new medicines for Gaucher disease in children

The European Medicines Agency and US Food and Drug Administration release joint proposal to facilitate clinical investigation of new medicines for Gaucher disease in children. The UK Gauchers Association will make a formal response to this draft joint proposal supporting this move. Tanya Collin-Histed and Jeremy Manuel in their roles for the European Gaucher Alliance (EGA) presented at the workshop in September 2012 (see ‘Joint Workshop’) link.